CN113164625B - 用于基因表达的突变体反向四环素反式激活因子 - Google Patents

用于基因表达的突变体反向四环素反式激活因子

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Publication number
CN113164625B
CN113164625B CN201980076921.7A CN201980076921A CN113164625B CN 113164625 B CN113164625 B CN 113164625B CN 201980076921 A CN201980076921 A CN 201980076921A CN 113164625 B CN113164625 B CN 113164625B
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nucleic acid
engineered nucleic
sequence
seq
promoter
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CN113164625A (zh
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D.A.辛克莱尔
Y.卢
N.J.戴维森
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Harvard University
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Harvard University
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Zoology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CN201980076921.7A 2018-09-28 2019-09-27 用于基因表达的突变体反向四环素反式激活因子 Active CN113164625B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862738894P 2018-09-28 2018-09-28
US62/738,894 2018-09-28
PCT/US2019/053492 WO2020069339A1 (en) 2018-09-28 2019-09-27 Mutant reverse tetracycline transactivators for expression of genes

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CN113164625A CN113164625A (zh) 2021-07-23
CN113164625B true CN113164625B (zh) 2026-04-14

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US (1) US20210403923A1 (https=)
EP (1) EP3856260A1 (https=)
JP (1) JP7589997B2 (https=)
CN (1) CN113164625B (https=)
CA (1) CA3113470A1 (https=)
WO (1) WO2020069339A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12274733B2 (en) 2018-09-28 2025-04-15 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
MX2021003663A (es) 2018-09-28 2021-05-28 Harvard College Reprogramacion celular para revertir el envejecimiento y promover la regeneracion de organos y tejidos.
WO2021091935A1 (en) * 2019-11-05 2021-05-14 Lonza Walkersville, Inc. Allogeneic t-cells and methods for production thereof
GB202103327D0 (en) * 2021-03-10 2021-04-21 Babraham Inst Novel method
US12467917B2 (en) 2021-03-16 2025-11-11 California Institute Of Technology Reconstructing human early embryogenesis in vitro with pluripotent stem cells
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
JP2025511883A (ja) * 2022-04-06 2025-04-16 プレジデント アンド フェローズ オブ ハーバード カレッジ 中枢神経系の加齢を逆行させること
CN119546324A (zh) * 2022-07-12 2025-02-28 南京瑞初医药有限公司 组合物及其使用方法
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN116004789B (zh) * 2022-08-01 2024-06-04 湖南家辉生物技术有限公司 一种Seckel综合征致病基因CEP152突变位点的应用及其诊断试剂
US12252518B2 (en) 2023-01-06 2025-03-18 Life Biosciences, Inc. Methods of treating non-arteritic anterior ischemic optic neuropathy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101302553A (zh) * 2008-05-30 2008-11-12 广州华峰生物科技有限公司 基于环介导等温扩增技术的结核分歧杆菌基因快速诊断试剂盒及其检测方法

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
AU746850B2 (en) * 1994-07-01 2002-05-02 Tet Systems Holding Gmbh & Co. Kg Tetracycline-regulated transcriptional modulaters
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
DE19851415B4 (de) * 1998-11-07 2010-11-04 Wolfgang Hillen Mutanter Transaktivator
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US20030186281A1 (en) * 2001-12-21 2003-10-02 Wolfgang Hillen Modified tetracycline repressor protein compositions and methods of use
GB0327384D0 (en) * 2003-11-25 2003-12-31 Queen Mary & Westfield College Gene therapy
JP5155176B2 (ja) * 2005-11-17 2013-02-27 ティーイーティー システムズ ゲーエムベーハー ウント ツェーオー． カーゲー 誘導発現系
WO2008051854A2 (en) * 2006-10-20 2008-05-02 Trustees Of Boston University A tunable genetic switch for regulating gene expression
US8080647B2 (en) * 2006-11-22 2011-12-20 Pioneer Hi Bred International Inc Tetracycline repressor and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2643459B1 (en) * 2010-11-24 2017-09-27 Takara Bio USA, Inc. Inducible expression system transcription modulators comprising a distributed protein transduction domain and methods for using the same
EP2710130B1 (en) * 2011-05-19 2022-06-29 Fundación Pública Andaluza Progreso Y Salud Highly inducible dual-promoter lentiviral tet-on system
CN102816793B (zh) * 2012-08-22 2014-09-10 陕西师范大学 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用
DK2898076T3 (en) * 2012-09-19 2018-06-14 Dsm Ip Assets Bv METHOD OF CELL MODIFICATION USING ESSENTIAL GENES AS MARKERS AND EVEN RECYCLING THESE
KR20150087321A (ko) * 2012-11-21 2015-07-29 메르츠 파마 게엠베하 운트 코. 카가아 보툴리눔 신경독소 생물학적 활성의 결정을 위한 수단 및 방법
WO2014152607A2 (en) * 2013-03-14 2014-09-25 University Of Massachusetts Dosage compensating transgenes and cells
EP3298141A4 (en) * 2015-05-17 2019-01-09 The Medical Research, Infrastructure, And Health Services Fund Of The Tel Aviv Medical Center COMPOSITIONS AND METHOD FOR THE TREATMENT OF CANCER
AU2016315703A1 (en) * 2015-08-31 2018-03-08 The Trustees Of The University Of Pennsylvania Chimeric AAV-anti-VEGF for treating cancer in canines
WO2019023680A1 (en) * 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019099552A1 (en) * 2017-11-14 2019-05-23 The J. David Gladstone Institutes Methods of generating retinal pigment epithelium (rpe)

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101302553A (zh) * 2008-05-30 2008-11-12 广州华峰生物科技有限公司 基于环介导等温扩增技术的结核分歧杆菌基因快速诊断试剂盒及其检测方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Improvement of the reverse tetracycline transactivator by single amino acid substitutions that reduce leaky target gene expression to undetectable levels";Ian J Roney et al.;《Sci Rep》;第6卷;摘要、第2-6页结果部分、第6-7页方法部分、图1-3 *
Tight regulation of transgene expression by tetracycline-dependent activator and repressor in brain;S Uchida et al.;《Genes Brain Behav》;第5卷(第1期);摘要、第97-99页材料与方法部分、图4 *

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US20210403923A1 (en) 2021-12-30
JP2022501060A (ja) 2022-01-06
WO2020069339A8 (en) 2020-05-07
WO2020069339A1 (en) 2020-04-02
JP7589997B2 (ja) 2024-11-26
CA3113470A1 (en) 2020-04-02
EP3856260A1 (en) 2021-08-04
CN113164625A (zh) 2021-07-23

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